Disruptive technology and hemophilia care: The multiple impacts of emicizumab. Issue 4 (7th May 2021)

• Record Type: Journal Article
• Authors: Hermans, Cedric; Makris, Mike
• Is Part Of: Research and practice in thrombosis and haemostasis. Volume 5:Issue 4(2021)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2021-05-07
• Subjects: care -- disruptive technology -- emicizumab -- hemophilia -- inhibitor -- nonreplacement therapy; Thrombosis -- Periodicals; Hemostasis -- Periodicals; 616.135005
• DOI: 10.1002/rth2.12508
• Languages: English
• ISSNs: 2475-0379

Abstract

Abstract: Emicizumab, a bispecific antibody mimicking the action of factor VIII (FVIII), is currently the first and only approved and increasingly accessible disruptive treatment option for hemophilia A, a disease so far mainly treated with frequent intravenous infusions of FVIII concentrates or bypassing agents in case of inhibitor development. Other disruptive treatments are expected to follow, such as agents that rebalance coagulation and gene therapy with the ambition of curing hemophilia. While these treatment options represent major achievements or expectations, their adoption and implementation should consider their multiple direct and indirect, immediate or delayed, consequences on hemophilia care globally. It is these multiple changes, present and future, already visible or hypothetical, that this article intends to review and explore.